A Recap of 2021 & Our Focus for 2022

2021 was an eventful and impactful year for regulatory policy. In such turbulent times, Biocom California worked to support members navigating the ever-changing landscape of regulation and provide up-to-date pertinent information. We look forward to continuing to provide updates and insight as members navigate even more changes to come in 2022.

Regulatory Highlights & Accomplishments from 2021

► We created a Cell & Gene Therapy Working Group – now the Cell & Gene Therapy Committee – comprised of 70+ members who convened five times in 2021 to discuss the future of cell & gene therapy, CIRM funding, reimbursement models, and more.

► We submitted five comment letters on behalf of our members:

► We hosted three industry events with the FDA: Diversity in Clinical Trials, The Digital Health Center of Excellence, and The Joint Regulation of Cell-Based Meat and Dairy Products by FDA and USDA.

► We hosted Regulatory Affairs Committee meetings on post-market surveillance, the drug supply chain and reporting, and welcomed Jesse Goodman, former Chief Scientist at the FDA and CBER Director, to discuss communicating with FDA during crisis times. 

Thank you to everyone who joined events, shared comments, and collaborated to form our new committee. 

On the Horizon for 2022

CDRH, CBER, and CDER issued annual agendas for their target draft and final guidances; CDRH at the start of the federal fiscal year, and CBER and CDER most recently at the start of the calendar year. Click here for a deeper dive into these agendas.

CDRH’s agendas list two key guidances that Biocom California plans to comment on: the EUA transition plans and enforcement discretion plans. A key draft guidance on remanufacturing (which Biocom California commented on in 2021) is likely to be finalized as well. 

CBER plans to focus heavily on cell and gene therapy, with planned draft guidances on the development of CAR-T therapies and human gene therapies and more on the regulation and evaluation of tissue-based products.

CDER’s draft guidances cover a range of issues with clinical trials and generics being  key areas for the center. In particular, CDER’s draft guidances plan to outline how clinical trials can be decentralized and how they can increase the use of real-world evidence, an area that PDUFA also plans to improve. 

Request for Comments

Draft Guidance on EUA Transition
On December 23rd, FDA released the highly anticipated Transition Plan for Medical Devices Authorized under the Emergency Use Authorization (EUA). 

Health and Human Services (HHS) has not terminated the EUA declaration and FDA outlines that it would come with an 180-day notice. FDA wants to facilitate an orderly transition and does not plan to pull products from the market during the transition time. FDA seeks guidance on whether the 180-day period is enough to transition products without causing issues (particularly in supply chain). Secondly, FDA wants feedback on whether this guidance meets the statutory requirement for consultation (that comes with an EUA authorization), given the volume of products with an EUA that would need to transition and FDA’s already stretched resources. 

Biocom California will submit comments on this draft guidance by the March 23rd deadline. Please contact Isabel Omer with feedback and comments on the draft by February 28th.

Key Issue Updates

CMS Restricts Coverage for Monoclonal Antibodies for Treatment of Alzheimer’s
On January 11th, the Centers for Medicare & Medicaid Services (CMS) released a proposed National Coverage Determination (NCD) decision memorandum. The proposed NCD would cover FDA-approved monoclonal antibodies that target amyloid for the treatment of Alzheimer’s disease through coverage with evidence development (CED) – which means that FDA-approved drugs in this class would be covered for Medicare beneficiaries only if they are enrolled in qualifying clinical trials. 

This decision comes after mixed reactions from agency officials and Congress to the approval of aduhelm under the accelerated approval pathway at FDA. The decision restricts coverage of these antibodies and risks setting a standard for CMS not covering products, despite FDA approval. 

OSHA Withdraws Emergency Temporary Standard
As of January 26th, US Department of Labor’s Occupational Safety and Health Administration (OSHA) announced they would withdraw the emergency temporary standard (ETS) for vaccination and testing of employees of businesses with 100 or more employees. This comes after the Supreme Court stayed the ETS. 
While OSHA is withdrawing the emergency temporary standard (ETS), they are not withdrawing the proposed rule. This may lead to continued efforts to require vaccination in large companies from the federal level. 

House Health Subcommittee Holds Hearing on PDUFA
On February 3rd, the Subcommittee on Health of the Committee on Energy and Commerce held a hearing entitled “FDA User Fee Reauthorization: Ensuring Safe and Effective Drugs and Biologics.” The hearing served as an important first step in the Congressional process to reauthorize the User Fee Agreements that permit the Food and Drug Administration (FDA) to collect fees, that supplement normal appropriations, in order to facilitate the timely review of a product. The hearing encompassed the Prescription Drug User Fee Act (PDUFA), the Biosimilar User Fee Act (BsUFA), and Generic Drug User Fee Amendments (GDUFA) agreements that were transmitted to Congress in January. Key provisions from each of the agreements can be found in the Chairman’s memo. 

During the hearing, many of the remarks made by Members of Congress and the FDA were supportive of the User Fee process and the negotiated agreement. The FDA is largely funded by user fees (especially CDER and CBER) and without reauthorization, the FDA operations and employees funded by user fees (which includes about 5,000 or 80 percent of the medical product review staff) would be in jeopardy.

Members of Congress noted several areas of priority, including: reviewing the accelerated approval pathway (particularly the withdrawal process for a drug); supply chain concerns for drug development; and an increased push for cell and gene therapies and rare disease research.